Standardisation enables the adoption of new technologies used in the manufacturing and testing of emerging therapies. In this presentation, we will share information on standards-setting activities at the United States Pharmacopeia (USP) and the type of tools and solutions to support implementing quality principles from early stages of cell therapy development, to manufacturing, and release of these products to patients. Cases studies on recently released standards, will be discussed.

Compared to other medicinal products, cell therapy products (including gene-modified) tend to use a lot of biological raw materials. These can be human, animal, microbial or even plant-derived. Without understanding how these materials are made, it is not possible to ensure their adventitious agent risks are addressed. Using real examples, this talk will discuss the princlples and how to assess and mitigate the identified risks.

Cell therapies are transforming the way diseases are treated. This presentation will discuss current challenges in cell therapy CMC optimization and how digital technologies are playing a key role.

Process characterization is a critical phase of process development supported by regulatory guidance for biologics development. Its experimental work plan aims at defining the design space for the process, secure a robust execution and ensure constant product quality. It is especially critical for cell therapies in general and is all the more crucial as they are now transitioning from the world of grafts, where they have grown for decades, into that of off-the-shelf pharmaceutical products. Cellectis pioneered the development and clinical use of allogeneic off-the-shelf CAR-T cells products to treat cancers. This approach is supported by a process platform where individual unit operations are assembled for each process/product version. Our pragmatic approach to characterize the UCART22-P2 process leveraged the deep knowledge gained over years and more than 2000 experiments. It helped locking production methods and control strategy very close to the original process flow. Each of the 260 operational parameters were investigated to determine their impact on product quality attributes and process performance. Critical Process Parameters (CPPs), Proven Acceptable Ranges (PARs) where then identified together with associated process changes. The work was undertaken with a platform approach paving the way for faster future studies supporting other products filling Cellectis’ pipeline.

As cell therapy products reach the market the conversation is changing from "can you manufacture it" to can you manufacture it suitable for millions of patients compared to hundreds. The role of local academic/hospital GMP centres which got the industry started, continues to be important but alternative solutions are needed for therapies to reach all patients who need them once they are approved.

Resolution Therapeutics is developing a macrophage cell therapy to treat inflammatory organ disease. This presentation will discuss the challenges and opportunities of implementing a decentralized manufacturing strategy in a phase appropriate manner and the associated benefits and risks in providing patients with access to quality advanced therapeutics

This presentation will highlight how POCare solutions leveraging the latest technology and data advances can overcome conventional CGT processing challenges and enable standardization. By working to develop closed, automated, and reproducible processes, it is possible to bring treatments closer to patients either at or within hours of local hospitals and treatment facilities. The mission is to decentralize and standardize production of these innovative therapies so that they are more affordable and accessible to patients across the globe and not just in major (or wealthier) markets.

The scale of cell therapy manufacturing operations, particularly autologous processes, brings new challenges and opportunities in the way that these therapies are produced. The traditional approach of having large-scale commercial manufacturing facilities brings fresh challenges in relation to CoGs and logistics considerations.  This creates opportunities for decentralised manufacturing which has significant potential and its own challenges with respect to ensuring suitably designed and regulatory compliant manufacturing areas.

Cell therapies require unique and complex starting material, production, and affordability supply chain challenges. This presentation will discuss innovative strategies being tested and employed to optimise the production and delivery of these therapies to patients in community settings—outside of large urban research centers—across the globe.

Optimisation of scale up manufacturing strategies for the production of cell-based therapies requires the establishment of robust criteria for elegibility of human origin starting materials and reliable bioprocess designs. In this presentation, we will present the strategy followed in our laboratory for the production of Mesenchymal Stromal Cells derived from the Wharton's jelly of the umbilical cord. Our approach involves the use of microcarriers and single use bioreactors.

Chris Crowell, Kite's vice president of operations, site head EU manufacturing and managing director provides his insights and perspectives into unique processes and challenges in the manufacturing of cell therapies; Kite’s manufacturing network and delivery of cell therapies to eligible patients across Europe and the world; and the importance of site qualification and teamwork throughout the full cell journey.

Chris sits down 1:1 with moderator Lee Buckler to share his personal experiences and advice focusing on: (1) Building on existing capabilities to meet increasing patient demand; (2) Delivering innovative therapies in Europe - barriers to access CAR T-cell therapy; (3) The next chapter in commercializing cell therapies.

- Success stories and current issues

- Novel approaches, new technologies

- Time-to-clinics

- Cost of manufacturing

- Batch-to-batch consistency

- Quality and patient safety

Gene and cell therapy are gaining more and more importance in efficient personalized precision medicine. From a manufacturing aspect, transient production of Lentiviral vectors for gene and cell therapeutics is fairly demanding in requiring chemical gene synthesis, plasmid DNA fermentation and purification, viral vector design and production via mammalian cell cultures such as HEK 293 cells, isolation of autologous human T-cells from the patient or engineering of allogenic T-cells, propagation of the T-cells and transfection with the viral vector carrying the gene of interest for a ligand on the T-cell surface targeting the T-cell to the tumor cell to express their cytotoxic effect. A short cut to Lentiviral vector transfection of T-Cells are stable HEK 293 packaging cell lines for explorative research of several transforming plasmids and stable HEK 293 producer cell lines for Phase III clinical trials and commercial manufacturing of autologous or allogenic T-Cells.An End-to-End CDMO for all required manufacturing technologies is advisable and facilitate the process chain if inhouse capabilities are limited.

Cryopreservation of iPS cells in large numbers or of functional, differentiated cells is also crucial for later success in therapy or other applications. Therefore, to meet the increasing demand in downstream applications, we have established robust, scalable cryopreservation protocols for iPS cells and several derivatives. This includes the use of large volume cryobags, ready-to-use formats and cryopreservation of tissue engineered products

Cell therapy is a promising, rapidly advancing field with the potential to transform medicine across disease areas where significant need exists. This presentation will highlight the array of cutting-edge technologies Astra Zeneca is implementing to drive important advances in cell therapy, including gene editing to create ‘universal cells’ that can be given to any patient, and antibodies to target our cell therapies where they are needed most.